GANX Gain Therapeutics Inc.

6.95
-0.19  -3%
Previous Close 7.14
Open 7.1
52 Week Low 6.5
52 Week High 17.93
Market Cap $82,542,648
Shares 11,876,640
Float 10,783,957
Enterprise Value $39,360,444
Volume 21,320
Av. Daily Volume 175,461
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Drug Pipeline

Drug Stage Notes
GT-02287, GT-02329
Gaucher and Parkinson's Disease
Phase 1
Phase 1
Preclinical positive topline data released September 8, 2021. IND-enabling studies initiation anticipated in 2022.

Latest News

  1. BETHESDA, Md., Oct. 19, 2021 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (NASDAQ:GANX) ("Gain", or the "Company"), a biotechnology company focused on identifying and optimizing allosteric binding sites never before targeted in neurodegenerative diseases and lysosomal storage disorders, today announced the appointment of Matthias Alder as Chief Operating Officer to advance the Company's strategic and operational objectives, including the establishment of additional industry partnerships and the transition of the Company's lead program in Gaucher and Parkinson's Disease into clinical trials, which is expected to occur in 2022.

    "I am delighted to welcome Matthias to Gain's growing team as we methodically scale our platform and build our internal…

    BETHESDA, Md., Oct. 19, 2021 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (NASDAQ:GANX) ("Gain", or the "Company"), a biotechnology company focused on identifying and optimizing allosteric binding sites never before targeted in neurodegenerative diseases and lysosomal storage disorders, today announced the appointment of Matthias Alder as Chief Operating Officer to advance the Company's strategic and operational objectives, including the establishment of additional industry partnerships and the transition of the Company's lead program in Gaucher and Parkinson's Disease into clinical trials, which is expected to occur in 2022.

    "I am delighted to welcome Matthias to Gain's growing team as we methodically scale our platform and build our internal clinical trial capabilities and operations," said Eric Richman, Chief Executive Officer of Gain. "He brings extensive experience in the pharma and biotech industries and is a seasoned executive who will complement our current executive team at a pivotal time for Gain as we grow the organization and prepare to advance our lead program into clinical trials."

    Matthias Alder joins Gain with more than 25 years of transactional, operational, and business development experience in the pharmaceutical and biotechnology industries. Most recently, Mr. Alder was Chief Business Officer at Autolus Therapeutics, a biotechnology company focused on developing CAR T-cell therapies for hematological and solid tumors, where he led the corporate development, legal, IP and HR functions. Prior to joining Autolus, he served as EVP of Business Development & Licensing and General Counsel of Sucampo Pharmaceuticals. During his tenure there, Mr. Alder established a late-stage development pipeline in orphan and rare CNS and oncology indications through acquisitions and strategic alliances. He has also held executive management positions at Cytos Biotechnology AG as EVP Corporate Development and General Counsel, and Micromet, Inc. as SVP Administration and General Counsel. Earlier in his career, Matthias was a partner in the Life Sciences Transactions Practice at Cooley LLP and in-house counsel for Novartis' pharmaceutical business.

    "I am excited to join Gain Therapeutics and work with Eric and the team to advance and expand the pipeline of drug candidates in CNS, oncology and other therapeutic areas through internal programs and partnerships," said Matthias Alder. "Using its supercomputer-powered SEE-Tx™ discovery platform, Gain has already established a track record in rapid and successful drug discovery and early development and is poised to make a real difference in the clinic for patients with Parkinson's and other neurodegenerative diseases."

    About Gain Therapeutics, Inc.

    Gain Therapeutics, Inc. is positioned at the confluence of technology and healthcare and focused on redefining drug discovery with its SEE-Tx™ target identification platform. By identifying and optimizing allosteric binding sites that have never before been targeted, Gain is unlocking new treatment options for difficult-to-treat disorders characterized by protein misfolding. Gain was established in 2017 with the support of its founders and institutional investors. It has been awarded funding support from The Michael J. Fox Foundation for Parkinson's Research (MJFF) and The Silverstein Foundation for Parkinson's with GBA, as well as from the Eurostars-2 joint program with co-funding from the European Union Horizon 2020 research and Innosuisse. In July 2020, Gain Therapeutics, Inc. completed a share exchange with Gain Therapeutics, SA, a Swiss corporation, whereby GT Gain Therapeutics SA became a wholly owned subsidiary of Gain Therapeutics, Inc.

    For more information, please visit https://www.gaintherapeutics.com

    About SEE-Tx™ and STARs

    The Company uses its proprietary supercomputer-based platform, Site-Directed Enzyme Enhancement Therapy ("SEE-Tx™"), to discover novel allosteric sites on misfolded proteins and identify proprietary small molecules (Structurally Targeted Allosteric Regulators, or "STARs") that bind these sites and restore protein folding, potentially treating the underlying disease. These small molecule binding sites, away from the protein's active areas, are called allosteric sites. The Company believes targeting the allosteric binding site instead of the active binding site can provide a number of advantages: superior regulation of misfolded proteins implicated in disease, enhanced specificity by being non-competitive with the natural substrate and the potential for molecules with favorable drug-like properties. Data obtained to date indicate that STARs can be administered orally and reach organs and tissues, including the brain, bone, and cartilage that are not typically accessible through current therapeutic options. In addition, the Company believes that STARs are relatively easy and cost-effective to manufacture.

    Forward-Looking Statements

    Any statements in this release that are not historical facts may be considered to be "forward-looking statements." Forward-looking statements are based on management's current expectations and are subject to risks and uncertainties which may cause results to differ materially and adversely from the statements contained herein. Such statements include, but are not limited to, statements regarding the market opportunity for Gain's product candidates, the business strategies and development plans of Gain, and the timing of preclinical and clinical studies. Some of the potential risks and uncertainties that could cause actual results to differ from those expected include Gain's ability to: make commercially available its products and technologies in a timely manner or at all; enter into strategic alliances, including arrangements for the development and distribution of its products; obtain intellectual property protection for its assets; accurately estimate and manage its expenses and cash burn and raise additional funds when necessary. Undue reliance should not be placed on forward-looking statements, which speak only as of the date they are made. Except as required by law, Gain does not undertake any obligation to update any forward-looking statements to reflect new information, events or circumstances after the date they are made, or to reflect the occurrence of unanticipated events.

    Investor & Media Contacts:

    Gain Therapeutics Investor Contact:

    Daniel Ferry

    LifeSci Advisors

    +1 (617) 430-7576

    daniel@lifesciadvisors.com

    Gain Therapeutics Media Contact:

    Joleen Schultz

    Joleen Schultz & Associates

    +1 760-271-8150

    joleen@joleenschultzassociates.com



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  2. BETHESDA, Md., Sept. 23, 2021 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (NASDAQ:GANX) ("Gain", or the "Company"), a biotechnology company focused on identifying and optimizing allosteric binding sites never before targeted in neurodegenerative diseases and lysosomal storage disorders, today announced that Eric Richman, Chief Executive Officer of Gain Therapeutics, will present at the Cantor Virtual Global Healthcare Conference and invites investors to participate via webcast and in one-on-one meetings. Please see additional details below:

    Cantor Virtual Global Healthcare Conference
     Date:Thursday, September 30th
     Time:8:00 to 8:30 a.m. ET
     Presenter:Eric Richman, Chief Executive Officer
     Webcast:Registration Link – Click Here
      * a replay will

    BETHESDA, Md., Sept. 23, 2021 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (NASDAQ:GANX) ("Gain", or the "Company"), a biotechnology company focused on identifying and optimizing allosteric binding sites never before targeted in neurodegenerative diseases and lysosomal storage disorders, today announced that Eric Richman, Chief Executive Officer of Gain Therapeutics, will present at the Cantor Virtual Global Healthcare Conference and invites investors to participate via webcast and in one-on-one meetings. Please see additional details below:

    Cantor Virtual Global Healthcare Conference

     Date:Thursday, September 30th
     Time:8:00 to 8:30 a.m. ET
     Presenter:Eric Richman, Chief Executive Officer
     Webcast:Registration Link – Click Here
      * a replay will be available following the presentation for 90 days

    Please contact your representative at Cantor Fitzgerald to schedule a virtual one-on-one meeting with Gain Therapeutics during the respective conference.

    For more information about the Cantor Virtual Global Healthcare Conference, please refer to the Cantor Fitzgerald conference website.

    About Gain Therapeutics, Inc.

    Gain Therapeutics, Inc. is positioned at the confluence of technology and healthcare and focused on redefining drug discovery with its SEE-Tx™ target identification platform. By identifying and optimizing allosteric binding sites that have never before been targeted, Gain is unlocking new treatment options for difficult-to-treat disorders characterized by protein misfolding. Gain was established in 2017 with the support of its founders and institutional investors. It has been awarded funding support from The Michael J. Fox Foundation for Parkinson's Research (MJFF) and The Silverstein Foundation for Parkinson's with GBA, as well as from the Eurostars-2 joint program with co-funding from the European Union Horizon 2020 research and Innosuisse. In July 2020, Gain Therapeutics, Inc. completed a share exchange with Gain Therapeutics, SA, a Swiss corporation, whereby GT Gain Therapeutics SA became a wholly owned subsidiary of Gain Therapeutics, Inc. For periods and at dates prior to the Corporate Reorganization, the consolidated financial statements were prepared based on the historical financial statements of GT Gain Therapeutics SA.



    For more information, please visit https://www.gaintherapeutics.com

    Forward-Looking Statements

    Any statements in this release that are not historical facts may be considered to be "forward-looking statements." Forward-looking statements are based on management's current expectations and are subject to risks and uncertainties which may cause results to differ materially and adversely from the statements contained herein. Such statements include, but are not limited to, statements regarding the market opportunity for Gain's product candidates, and the business strategies and development plans of Gain. Some of the potential risks and uncertainties that could cause actual results to differ from those expected include Gain's ability to: make commercially available its products and technologies in a timely manner or at all; enter into strategic alliances, including arrangements for the development and distribution of its products; obtain intellectual property protection for its assets; accurately estimate and manage its expenses and cash burn and raise additional funds when necessary. Undue reliance should not be placed on forward-looking statements, which speak only as of the date they are made. Except as required by law, Gain does not undertake any obligation to update any forward-looking statements to reflect new information, events or circumstances after the date they are made, or to reflect the occurrence of unanticipated events.

    Investor & Media Contacts:

    Gain Therapeutics Investor Contact:

    Daniel Ferry

    LifeSci Advisors

    +1 (617) 430-7576

    daniel@lifesciadvisors.com

    Gain Therapeutics Media Contact:

    Joleen Schultz

    Joleen Schultz & Associates

    +1 760-271-8150

    joleen@joleenschultzassociates.com



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  3. - Conference Dates: September 20th-23rd -

    BETHESDA, Md., Sept. 17, 2021 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (NASDAQ:GANX) ("Gain", or the "Company"), a biotechnology company focused on identifying and optimizing allosteric binding sites never before targeted in neurodegenerative diseases and lysosomal storage disorders, today announced that Eric Richman, Chief Executive Officer of Gain Therapeutics, will present at the Oppenheimer Fall Healthcare Life Sciences & MedTech Summit and invites investors to participate via webcast and in one-on-one meetings. Please see additional details below:

    Oppenheimer Fall Healthcare Life Sciences & MedTech Summit
    Date:Wednesday, September 22nd
    Time: 1:15 to 1:55 p.m. ET
    Presenter: Eric Richman, Chief

    - Conference Dates: September 20th-23rd -

    BETHESDA, Md., Sept. 17, 2021 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (NASDAQ:GANX) ("Gain", or the "Company"), a biotechnology company focused on identifying and optimizing allosteric binding sites never before targeted in neurodegenerative diseases and lysosomal storage disorders, today announced that Eric Richman, Chief Executive Officer of Gain Therapeutics, will present at the Oppenheimer Fall Healthcare Life Sciences & MedTech Summit and invites investors to participate via webcast and in one-on-one meetings. Please see additional details below:

    Oppenheimer Fall Healthcare Life Sciences & MedTech Summit
    Date:Wednesday, September 22nd
    Time: 1:15 to 1:55 p.m. ET
    Presenter: Eric Richman, Chief Executive Officer
    Webcast: Registration Link – Click Here
     * a replay will be available following the presentation for 90 days

    Please contact your representative at Oppenheimer & Co. to schedule a virtual one-on-one meeting with Gain Therapeutics during the respective conference.

    For more information about the Oppenheimer Fall Healthcare Life Sciences & MedTech Summit, please refer to the conference website.

    About Gain Therapeutics, Inc.

    Gain Therapeutics, Inc. is positioned at the confluence of technology and healthcare and focused on redefining drug discovery with its SEE-Tx™ target identification platform. By identifying and optimizing allosteric binding sites that have never before been targeted, Gain is unlocking new treatment options for difficult-to-treat disorders characterized by protein misfolding. Gain was established in 2017 with the support of its founders and institutional investors. It has been awarded funding support from The Michael J. Fox Foundation for Parkinson's Research (MJFF) and The Silverstein Foundation for Parkinson's with GBA, as well as from the Eurostars-2 joint program with co-funding from the European Union Horizon 2020 research and Innosuisse. In July 2020, Gain Therapeutics, Inc. completed a share exchange with Gain Therapeutics, SA, a Swiss corporation, whereby GT Gain Therapeutics SA became a wholly owned subsidiary of Gain Therapeutics, Inc. For periods and at dates prior to the Corporate Reorganization, the consolidated financial statements were prepared based on the historical financial statements of GT Gain Therapeutics SA.



    For more information, please visit https://www.gaintherapeutics.com

    Forward-Looking Statements

    Any statements in this release that are not historical facts may be considered to be "forward-looking statements." Forward-looking statements are based on management's current expectations and are subject to risks and uncertainties which may cause results to differ materially and adversely from the statements contained herein. Such statements include, but are not limited to, statements regarding the market opportunity for Gain's product candidates, and the business strategies and development plans of Gain. Some of the potential risks and uncertainties that could cause actual results to differ from those expected include Gain's ability to: make commercially available its products and technologies in a timely manner or at all; enter into strategic alliances, including arrangements for the development and distribution of its products; obtain intellectual property protection for its assets; accurately estimate and manage its expenses and cash burn and raise additional funds when necessary. Undue reliance should not be placed on forward-looking statements, which speak only as of the date they are made. Except as required by law, Gain does not undertake any obligation to update any forward-looking statements to reflect new information, events or circumstances after the date they are made, or to reflect the occurrence of unanticipated events.

    Investor & Media Contacts:

    Gain Therapeutics Investor Contact:

    Daniel Ferry

    LifeSci Advisors

    +1 (617) 430-7576

    daniel@lifesciadvisors.com

    Gain Therapeutics Media Contact:

    Joleen Schultz

    Joleen Schultz & Associates

    +1 760-271-8150

    joleen@joleenschultzassociates.com



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  4. BETHESDA, Md., Sept. 09, 2021 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (NASDAQ:GANX) ("Gain", or the "Company"), a biotechnology company focused on identifying and optimizing allosteric binding sites never before targeted in neurodegenerative diseases and lysosomal storage disorders, today announced that Manolo Bellotto, Ph.D., General Manager and President at Gain, will present an overview on the Company's proprietary Structurally Targeted Allosteric Regulators (STARs) for GM1 Gangliosidosis and Morquio B Disease.

    GM1 Virtual Community Conference

    Date:Tuesday, September 14th
    Time:2:00pm PT/5:00pm ET
    Presenter:Manolo Bellotto, Ph.D., General Manager and President

    To register to attend the conference visit https://registration.socio.events/e/gm1

    BETHESDA, Md., Sept. 09, 2021 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (NASDAQ:GANX) ("Gain", or the "Company"), a biotechnology company focused on identifying and optimizing allosteric binding sites never before targeted in neurodegenerative diseases and lysosomal storage disorders, today announced that Manolo Bellotto, Ph.D., General Manager and President at Gain, will present an overview on the Company's proprietary Structurally Targeted Allosteric Regulators (STARs) for GM1 Gangliosidosis and Morquio B Disease.

    GM1 Virtual Community Conference

    Date:Tuesday, September 14th
    Time:2:00pm PT/5:00pm ET
    Presenter:Manolo Bellotto, Ph.D., General Manager and President

    To register to attend the conference visit https://registration.socio.events/e/gm1

    Morquio B, also known as Mucopolysaccharidosis type IV (MPS IV), is a progressive disease mostly impacting the skeleton, caused by mutations in GLB1, the gene that encodes the beta-galactosidase (GLB) enzyme. GM1 Gangliosidosis is a hereditary, progressive disease mostly impacting neurons in the brain and spinal cord, caused by mutations in GLB1. In both diseases, these mutations result in the misfolding and subsequent dysfunction of GLB, which leads to toxic substrate accumulation in organs and tissues. Gain is developing allosteric regulators that are designed to decrease toxic substrate accumulation in organs and tissues of patients with Morquio B and GM1 Gangliosidosis in order to potentially provide the first treatment approach for the disease's neurological symptoms.

    About Gain Therapeutics, Inc.

    Gain Therapeutics, Inc. is positioned at the confluence of technology and healthcare and focused on redefining drug discovery with its SEE-Tx™ target identification platform. By identifying and optimizing allosteric binding sites that have never before been targeted, Gain is unlocking new treatment options for difficult-to-treat disorders characterized by protein misfolding. Gain was established in 2017 with the support of its founders and institutional investors. It has been awarded funding support from The Michael J. Fox Foundation for Parkinson's Research (MJFF) and The Silverstein Foundation for Parkinson's with GBA, as well as from the Eurostars-2 joint program with co-funding from the European Union Horizon 2020 research and Innosuisse. In July 2020, Gain Therapeutics, Inc. completed a share exchange with Gain Therapeutics, SA, a Swiss corporation, whereby GT Gain Therapeutics SA became a wholly owned subsidiary of Gain Therapeutics, Inc. For periods and at dates prior to the Corporate Reorganization, the consolidated financial statements were prepared based on the historical financial statements of GT Gain Therapeutics SA.

    For more information, please visit https://www.gaintherapeutics.com

    Forward-Looking Statements

    Any statements in this release that are not historical facts may be considered to be "forward-looking statements." Forward-looking statements are based on management's current expectations and are subject to risks and uncertainties which may cause results to differ materially and adversely from the statements contained herein. Such statements include, but are not limited to, statements regarding the market opportunity for Gain's product candidates, and the business strategies and development plans of Gain. Some of the potential risks and uncertainties that could cause actual results to differ from those expected include Gain's ability to: make commercially available its products and technologies in a timely manner or at all; enter into strategic alliances, including arrangements for the development and distribution of its products; obtain intellectual property protection for its assets; accurately estimate and manage its expenses and cash burn and raise additional funds when necessary. Undue reliance should not be placed on forward-looking statements, which speak only as of the date they are made. Except as required by law, Gain does not undertake any obligation to update any forward-looking statements to reflect new information, events or circumstances after the date they are made, or to reflect the occurrence of unanticipated events.

    Investor & Media Contacts:

    Gain Therapeutics Investor Contact:

    Daniel Ferry

    LifeSci Advisors

    +1 (617) 430-7576

    daniel@lifesciadvisors.com

    Gain Therapeutics Media Contact:

    Joleen Schultz

    Joleen Schultz & Associates

    +1 760-271-8150

    joleen@joleenschultzassociates.com



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  5. GT-02287and GT-02329 compounds display positive effects on GCase activity and related parameters
    --- 
    Study results demonstrate an increase in GCase protein levels with transport to the lysosomes and decrease of glucosylceramide and α-synuclein-p129 levels 
    --- 
    Comprehensive study results to be presented at Michael J. Fox Foundation's upcoming Innovating from Drug Discovery to the Clinic: Novel Approaches to PD Therapeutic Development webinar 
    --- 
    IND-initiating study planned for Q4'21

    BETHESDA, Md., Sept. 08, 2021 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (NASDAQ:GANX) ("Gain", or the "Company"), a biotechnology company focused on identifying and optimizing allosteric binding sites never before targeted in neurodegenerative diseases and lysosomal…

    GT-02287and GT-02329 compounds display positive effects on GCase activity and related parameters

    --- 

    Study results demonstrate an increase in GCase protein levels with transport to the lysosomes and decrease of glucosylceramide and α-synuclein-p129 levels 

    --- 

    Comprehensive study results to be presented at Michael J. Fox Foundation's upcoming Innovating from Drug Discovery to the Clinic: Novel Approaches to PD Therapeutic Development webinar 

    --- 

    IND-initiating study planned for Q4'21

    BETHESDA, Md., Sept. 08, 2021 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (NASDAQ:GANX) ("Gain", or the "Company"), a biotechnology company focused on identifying and optimizing allosteric binding sites never before targeted in neurodegenerative diseases and lysosomal storage disorders, today announced topline data from the Company's study, conducted at the University of Maryland School of Medicine (UMSOM). The study, evaluating two lead Structurally Targeted Allosteric Regulators (STARs) compounds – GT-02287 and GT-02329 – for the treatment of Gaucher and GBA1 Parkinson's Disease, demonstrated positive effects on all tested phenotypes.

    Ricardo Feldman, Ph.D., Associate Professor of Microbiology and Immunology at the University of Maryland School of Medicine, stated, "Our laboratory is using human induced pluripotent stem cells (iPSC) derived from patients with GD and GBA-associated Parkinson's disease to test the efficacy of the two lead STAR chaperones developed by Gain Therapeutics. Our studies in iPSC-derived cortical and dopaminergic neurons from neuronopathic Gaucher Disease patients show that these compounds increase the levels of GCase protein, its transport to the lysosome, and its enzymatic activity. In dopaminergic neurons, the two lead STAR chaperones also decrease the levels of α-synuclein-p129, demonstrating their potential to treat GBA1-associated Parkinson's Disease."

    "These data are extremely exciting, as it further demonstrates the potential of GT-02287 and GT-02329 and expands the body of evidence supporting our Site-Directed Enzyme Enhancement Therapy (SEE-Tx™) drug discovery platform," said Eric Richman, Chief Executive Officer of Gain. "We plan to fully evaluate the results of this study and present a complete data set on the upcoming

    Michael J. Fox Foundation's Innovating from Drug Discovery to the Clinic: Novel Approaches to PD Therapeutic Development webinar. In addition, we anticipate initiating IND-enabling studies for Gaucher / Parkinson's Disease in the fourth quarter of this year."

    Gain identified the two lead STAR candidates (GT-02287 and GT-02329) through its proprietary SEE-Tx™ platform. The compounds were characterized in assays to potentially help Parkinson's patients with GBA1 gene mutations as well as patients whose glucocerebrosidase (GCase) protein is misfolded due to ageing cellular processes. This previous work was supported through grants from The Michael J. Fox Foundation (MJFF) and the Silverstein Foundation for Parkinson's with GBA.

    Mutations in GBA1, the gene encoding the lysosomal enzyme GCase, are among the most commonly known genetic risk factors for the development of Parkinson's Disease and related synucleinopathies. GBA1 mutations are causative for the rare autosomal storage disorder Gaucher Disease and may lead to degradation of the protein, disruptions in lysosomal targeting and diminished performance of the enzyme in the lysosome. Patients with Gaucher Disease including heterozygous carriers are at increased risk of developing Parkinson's Disease and Dementia with Lewy Bodies.

    The study conducted in collaboration with Ricardo A. Feldman, Ph.D., Associate Professor of Microbiology and Immunology at UMSOM and lead investigator, is using human induced pluripotent stem cells (iPSC) for Gaucher and Parkinson's Disease modeling and drug discovery. Gain's candidate molecules GT-02287 and GT-02329 have been initially assessed in this unique "disease-in-a-dish" iPSC model derived from Gaucher type 2 and 3 patient cells.

    Topline data from collaboration with University of Maryland School of Medicine on GBA-associated Parkinson's Disease in iPSC model. The two STAR compounds achieved the following results:

    • Displayed positive effects on key biological read-outs such as GCase activity, GCase protein levels, GCase transport to the lysosomes, glucosylceramide and depletion of P-alpha-synuclein: all parameters that are related to disease onset and progression
    • Increased GCase enzyme activity
    • Increased GCase protein levels and transport to the lysosome in cortical neurons
    • Decreased GluCer accumulation in cortical neurons
    • Increased GCase protein levels and transport to the lysosome in dopaminergic neurons
    • Decreased α-synuclein-p129 levels in dopaminergic neurons

    "We appreciate the close collaboration with Dr. Feldman and the University of Maryland School of Medicine in this important area of research," said Manolo Bellotto, Ph.D., General Manager at Gain. "The topline data demonstrates that our STARs compounds open a new potential approach for direct treatment of GBA1 Parkinson's Disease by guiding misfolded forms of the GCase enzyme to their proper shape and restoring enzymatic activity. This is an exciting validation of our platform technology and a promising potential therapeutic opportunity for patients suffering from these debilitating diseases."

    The combined data from Gain Therapeutics and Dr. Feldman's laboratory suggest that STARs bind to novel allosteric binding sites identified using Gain's proprietary SEE-Tx™ drug discovery platform, stabilize, and restore enzymatic activity to misfolded GCase. In these studies, the restoration of GCase activity resulted in the depletion of the toxic phosphorylated and aggregated forms of α-synuclein and a reversal of the neurodegenerative process resulting in improved locomotor activity in Parkinson's Disease model in rats. Given that STARs are small molecule therapies, they have significant advantages over the current standard of care for disorders associated with protein misfolding, including most enzyme replacement therapies and gene therapies. They can be administered as a simple oral pill and can reach organs and tissues not typically accessible through current therapeutic options, including the brain, bone and cartilage. As well, the Company believes that STARs are relatively easy and cost-effective to manufacture.

    "These encouraging results show promise for this approach to correct dysfunction in the GBA1 pathway, a leading target for Parkinson's drug development," said Marco Baptista, PhD, MJFF Vice President of Research Programs. "We look forward to hearing more on next steps to advance these potential therapies further in testing and closer to patients whose greatest unmet need is a treatment to slow or stop disease progression."

    About Gain Therapeutics, Inc.

    Gain Therapeutics, Inc. is positioned at the confluence of technology and healthcare and focused on redefining drug discovery with its SEE-Tx™ target identification platform. By identifying and optimizing allosteric binding sites that have never before been targeted, Gain is unlocking new treatment options for difficult-to-treat disorders characterized by protein misfolding. Gain was established in 2017 with the support of its founders and institutional investors. It has been awarded funding support from The Michael J. Fox Foundation for Parkinson's Research (MJFF) and The Silverstein Foundation for Parkinson's with GBA, as well as from the Eurostars-2 joint program with co-funding from the European Union Horizon 2020 research and Innosuisse. In July 2020, Gain Therapeutics, Inc. completed a share exchange with Gain Therapeutics, SA, a Swiss corporation, whereby GT Gain Therapeutics SA became a wholly owned subsidiary of Gain Therapeutics, Inc. For periods and at dates prior to the Corporate Reorganization, the consolidated financial statements were prepared based on the historical financial statements of GT Gain Therapeutics SA.



    For more information, please visit https://www.gaintherapeutics.com

    About the University of Maryland School of Medicine

    Now in its third century, the University of Maryland School of Medicine was chartered in 1807 as the first public medical school in the United States. It continues today as one of the fastest growing, top-tier biomedical research enterprises in the world -- with 46 academic departments, centers, institutes, and programs, and a faculty of more than 3,000 physicians, scientists, and allied health professionals, including members of the National Academy of Medicine and the National Academy of Sciences, and a distinguished two-time winner of the Albert E. Lasker Award in Medical Research. With an operating budget of more than $1.2 billion, the School of Medicine works closely in partnership with the University of Maryland Medical Center and Medical System to provide research-intensive, academic and clinically based care for nearly 2 million patients each year. The School of Medicine has nearly $600 million in extramural funding, with most of its academic departments highly ranked among all medical schools in the nation in research funding. As one of the seven professional schools that make up the University of Maryland, Baltimore campus, the School of Medicine has a total population of nearly 9,000 faculty and staff, including 2,500 student trainees, residents, and fellows. The combined School of Medicine and Medical System ("University of Maryland Medicine") has an annual budget of over $6 billion and an economic impact of nearly $20 billion on the state and local community. The School of Medicine, which ranks as the 8th highest among public medical schools in research productivity (according to the Association of American Medical Colleges profile) is an innovator in translational medicine, with 606 active patents and 52 start-up companies. In the latest U.S. News & World Report ranking of the Best Medical Schools, published in 2021, the UM School of Medicine is ranked #9 among the 92 public medical schools in the U.S., and in the top 15 percent (#27) of all 192 public and private U.S. medical schools. The School of Medicine works locally, nationally, and globally, with research and treatment facilities in 36 countries around the world. Visit medschool.umaryland.edu. To learn more about Dr. Feldman's research visit https://www.medschool.umaryland.edu/profiles/Feldman-Ricardo/

    About The Michael J. Fox Foundation for Parkinson's Research

    As the world's largest nonprofit funder of Parkinson's research, The Michael J. Fox Foundation is dedicated to accelerating a cure for Parkinson's disease and improved therapies for those living with the condition today. The Foundation pursues its goals through an aggressively funded, highly targeted research program coupled with active global engagement of scientists, Parkinson's patients, business leaders, clinical trial participants, donors and volunteers. In addition to funding $1 billion in research to date, the Foundation has fundamentally altered the trajectory of progress toward a cure. Operating at the hub of worldwide Parkinson's research, the Foundation forges groundbreaking collaborations with industry leaders, academic scientists and government research funders; increases the flow of participants into Parkinson's disease clinical trials with its online tool, Fox Trial Finder; promotes Parkinson's awareness through high-profile advocacy, events and outreach; and coordinates the grassroots involvement of thousands of Team Fox members around the world. For more information, visit us on at https://www.michaeljfox.org.

    Forward-Looking Statements

    Any statements in this release that are not historical facts may be considered to be "forward-looking statements." Forward-looking statements are based on management's current expectations and are subject to risks and uncertainties which may cause results to differ materially and adversely from the statements contained herein. Such statements include, but are not limited to, statements regarding the market opportunity for Gain's product candidates, and the business strategies and development plans of Gain. Some of the potential risks and uncertainties that could cause actual results to differ from those expected include Gain's ability to: make commercially available its products and technologies in a timely manner or at all; enter into strategic alliances, including arrangements for the development and distribution of its products; obtain intellectual property protection for its assets; accurately estimate and manage its expenses and cash burn and raise additional funds when necessary. Undue reliance should not be placed on forward-looking statements, which speak only as of the date they are made. Except as required by law, Gain does not undertake any obligation to update any forward-looking statements to reflect new information, events or circumstances after the date they are made, or to reflect the occurrence of unanticipated events.

    Investor & Media Contacts:

    Gain Therapeutics Investor Contact:

    Daniel Ferry

    LifeSci Advisors

    +1 (617) 430-7576

    daniel@lifesciadvisors.com

    Gain Therapeutics Media Contact:

    Joleen Schultz

    Joleen Schultz & Associates

    +1 760-271-8150

    joleen@joleenschultzassociates.com



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